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Table 1 Characteristics of the indications (n = 78)

From: Clinical evidence for orphan medicinal products-a cause for concern?

  Number of indications (%)
Prevalence of the rare disease in which the indication is authorized
 Between 4/10 000 and 5/10 000 1 (1.3%)
 Between 3/10 000 and 4/10 000 3 (3.8%)
 Between 2/10 000 and 3/10 000 5 (6.4%)
 Between 1/10 000 and 2/10 000 14 (17.9%)
 Between 1/10 000 and 2/10 000 42 (53.8%)
Requested protocol assistance
 Yes 22 (28.2%)
 No 15 (19.2%)
 Not reported 41 (52.6%)
Performed dose finding study
 Yes 44 (56.4%)
 No 25 (32.0%)
 Not reported 9 (11.5%)